The U.S. Food and Drug Administration (FDA) has issued a positive evaluation of ORKAMBI (lumacaftor/ivacaftor) as a recommended treatment for cystic fibrosis (CF) patients 12 years and older with two copies of the F508del mutation in the CFTR gene. The treatment developed by Vertex Pharmaceuticals Incorporated was recommended in a 12 to 1 vote by the FDA’s Pulmonary-Allergy Drugs Advisory Committee (PADAC).
The FDA’s advisory committees give the agency independent scientific and medical recommendations regarding the safety, effectiveness and appropriate use of new medical products and, despite the fact that there is no tie between the committee’s advice and the FDA’s decision, it is an important indicator. The agency is expected to issue a final decision by July 5th, according to the Prescription Drug User Fee Act (PDUFA), as stated by the company in a press release.
Following an approval by the FDA, ORKAMBI may become the first and only drug combination to address the underlying cause for the development of CF in patients with two copies of the F508del mutation in the CFTR gene older than 12 years of age. This group represents a large proportion of CF patients, and the lumacaftor/ivacaftor drug combination may help about 8,500 people with medical characteristics consistent with the treatment.
“Today’s positive recommendation brings the cystic fibrosis community one step closer to potential approval of the first medicine to treat the underlying cause of this disease for many more people,” stated the executive vice president and chief medical officer at Vertex, Jeffrey Chodakewitz, MD. “We look forward to continuing to work with the FDA and other regulatory agencies throughout the world to make ORKAMBI available to eligible patients as soon as possible.”
Lumacaftor is a drug developed to improve the amount of functional protein at the surface of cells through the correction of both the process and traffic of protein defect. Ivacaftor, on the other hand, increases the CFTR protein function by reaching the cell surface. ORKAMBI, which combines the two in co-formulated tablets, is an oral medication.
Vertex announced last November the submission of a New Drug Application (NDA) to the FDA, as well as a Marketing Authorization Application (MAA) to the European Medicine’s Agency (EMA) for a fully co-formulated combination of lumacaftor (400mg q12h) and ivacaftor (250mg q12h). In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface.